Real-world experience with tofacitinib in ulcerative colitis: a systematic review and meta-analysis
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Laura A Lucaciu1, Nathan Constantine-Cooke2, Nikolas Plevris1
1Edinburgh IBD Unit, Western General Hospital, Edinburgh, UK.
Background And Aims:
Tofacitinib is a Janus kinase inhibitor (JAKi) recently approved for the treatment of moderate to severe ulcerative colitis (UC) based on robust efficacy and safety data derived from OCTAVE clinical trials. Evidence on the outcomes of tofacitinib therapy in real-world UC patients is needed, as a number of these patients would be deemed ineligible for clinical trials. We have therefore summarised data derived from observational, real-world evidence (RWE) studies on the effectiveness and safety of tofacitinib in moderate to severe UC patients.
Methods:
We searched the PubMed, EMBASE, Scopus, Web of Science and Cochrane databases for observational studies on the use of tofacitinib in UC patients, published between 30 May 2018 and 24 January 2021. Pooled induction (8-14 weeks) and maintenance (16-26 weeks) clinical response and remission rates were calculated, as well as the proportion of reported adverse events using random effects models.
Results:
Nine studies were included, comprising 830 patients, of which 81% were previously treated with anti-tumour necrosis factor (TNF) and 57% with vedolizumab. Induction of clinical response and remission were achieved in 51% (95% confidence interval, 41-60%) and 37% (26-45%) of patients, after a median follow-up of 8 weeks. At the end of a median follow-up of 24 weeks, maintenance of clinical response and remission were met in 40% (31-50%) and 29% (23-36%) of patients, respectively. Thirty-two percent of the patients had at least one adverse event, the most commonly reported being mild infection (13%) and worsening of UC, requiring colectomy (13%). A third of the patients (35%) discontinued tofacitinib, most frequently due to primary non-response (51%).
Conclusion:
Tofacitinib is a safe and effective therapy in real-world UC patients, as previously reported by clinical trials.
Insights
Real-world data show tofacitinib is effective for ulcerative colitis (UC) patients, achieving clinical response and remission. Tofacitinib is a safe treatment option for moderate to severe UC, with manageable adverse events.
Area of Science:
- Gastroenterology
- Immunology
- Pharmacology
Background:
- Tofacitinib, a Janus kinase inhibitor (JAKi), is approved for moderate to severe ulcerative colitis (UC).
- Real-world evidence (RWE) is needed to assess tofacitinib's effectiveness and safety in patients not eligible for clinical trials.
Purpose of the Study:
- To summarize real-world evidence on the effectiveness and safety of tofacitinib in moderate to severe UC patients.
- To evaluate pooled induction and maintenance response and remission rates, and adverse event proportions.
Main Methods:
- A systematic literature search was conducted across major databases (PubMed, EMBASE, Scopus, Web of Science, Cochrane).
- Observational studies published between May 2018 and January 2021 were included.
- Random effects models were used to calculate pooled rates for clinical response, remission, and adverse events.
Main Results:
- Nine studies comprising 830 patients were analyzed; 81% had prior anti-TNF therapy and 57% prior vedolizumab.
- Induction of clinical response and remission were achieved in 51% and 37% of patients, respectively.
- Maintenance of response and remission were observed in 40% and 29% of patients. Adverse events occurred in 32%, with mild infections and colectomy being most common. Discontinuation was 35%, primarily due to non-response.
Conclusions:
- Tofacitinib demonstrates safety and effectiveness in real-world UC patient populations.
- These findings align with previous clinical trial results, supporting tofacitinib's role in UC management.
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